Cardiac Medication Use in ACTION for Duchenne Muscular Dystrophy Cardiomyopathy

This study seeks to understand cardiac medication use in a large cohort of males with Duchenne Muscular Dystrophy (DMD) followed prospectively with focus on current practices and adherence to consensus directed medical therapy (CDMT). DMD patients have been enrolled in the Advanced Cardiac Therapies Improving Outcomes Network (ACTION) Dystrophinopathy Registry since 2021. Cardiac medication use was analyzed at enrollment and most recent follow-up. CDMT was defined as concurrent use of angiotensin-converting-enzyme inhibitor (ACEi)/angiotensin II receptor blocker (ARB) /angiotensin receptor-neprilysin inhibitor (ARNI) plus beta-blocker (BB) plus mineralocorticoid receptor antagonist (MRA). Two hundred sixty-five males with DMD (median age 17.5 (IQR 14.5-21.5) years) were prospectively followed; median follow-up was 11.5 (IQR 6.2-15.6) months. At most recent follow-up, 153 patients (57.7%) had decreased LV systolic function, 67 (25.3% of the cohort) had moderate or severe dysfunction. For patients with moderate or severe dysfunction, CDMT was used for 49/67 (73.1%) at most recent follow-up, similar to 36/51 (70.6%) at enrollment (p = 0.92). Target doses of CDMT were achieved for 27% of males on ACEi/ARB/ARNI, 28% on BB, and 23% on MRA. Initial analysis of a prospective registry of males with DMD showed that ~ 30% of patients with moderate or severe LV dysfunction were not on CDMT at most recent follow-up and the majority did not reach target dosing. Further understanding regarding the optimal combination of cardiac medications for DMD cardiomyopathy is needed, as is a better understanding of the barriers to CDMT optimization given increasing cardiac causes of death for DMD patients.

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Publication Details

Journal
Pediatric Cardiology
Published
2025-06-20
DOI
https://doi.org/10.1007/s00246-025-03917-2
Citations
3
Primary Topic
Muscle Physiology and Disorders
Type
article
Field-Weighted Citation Impact
2.42
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article

Cardiac Medication Use in ACTION for Duchenne Muscular Dystrophy Cardiomyopathy

Daphne T. Hsu, Deipanjan Nandi, Carol Wittlieb‐Weber, Katheryn Gambetta et al.
3 citations
Pediatric Cardiology
Muscle Physiology and Disorders
2.42
article

Cardiac Medication Use in ACTION for Duchenne Muscular Dystrophy Cardiomyopathy

Daphne T. Hsu, Deipanjan Nandi, Carol Wittlieb‐Weber, Katheryn Gambetta, Bethany Wisotzkey, Chet Villa, Chesney Castleberry, Deepa Mokshagundam, Frank J. Raucci, Sarah Wilkens, Renata Shih, Jonathan H. Soslow, Tyler W. Cunningham, Angela Lorts, Nelia Soares, Brian Birnbaum, Jennifer Conway, Emily A. Hayes, Ashwin K. Lal, Paul Esteso, Beth D. Kaufman, John J. Parent, Svetlana Shugh, Hugo R. Martinez, B. Kroslowitz
article en
3 citations

Abstract

This study seeks to understand cardiac medication use in a large cohort of males with Duchenne Muscular Dystrophy (DMD) followed prospectively with focus on current practices and adherence to consensus directed medical therapy (CDMT). DMD patients have been enrolled in the Advanced Cardiac Therapies Improving Outcomes Network (ACTION) Dystrophinopathy Registry since 2021. Cardiac medication use was analyzed at enrollment and most recent follow-up. CDMT was defined as concurrent use of angiotensin-converting-enzyme inhibitor (ACEi)/angiotensin II receptor blocker (ARB) /angiotensin receptor-neprilysin inhibitor (ARNI) plus beta-blocker (BB) plus mineralocorticoid receptor antagonist (MRA). Two hundred sixty-five males with DMD (median age 17.5 (IQR 14.5-21.5) years) were prospectively followed; median follow-up was 11.5 (IQR 6.2-15.6) months. At most recent follow-up, 153 patients (57.7%) had decreased LV systolic function, 67 (25.3% of the cohort) had moderate or severe dysfunction. For patients with moderate or severe dysfunction, CDMT was used for 49/67 (73.1%) at most recent follow-up, similar to 36/51 (70.6%) at enrollment (p = 0.92). Target doses of CDMT were achieved for 27% of males on ACEi/ARB/ARNI, 28% on BB, and 23% on MRA. Initial analysis of a prospective registry of males with DMD showed that ~ 30% of patients with moderate or severe LV dysfunction were not on CDMT at most recent follow-up and the majority did not reach target dosing. Further understanding regarding the optimal combination of cardiac medications for DMD cardiomyopathy is needed, as is a better understanding of the barriers to CDMT optimization given increasing cardiac causes of death for DMD patients.

Pediatric CardiologyVol. 47(4)
Indiana University Health (US), Cincinnati Children's Hospital Medical Center (US), Boston Children's Hospital (US), Lurie Children's Hospital (US), Seattle Children's Hospital (US), Monroe Carell Jr. Children's Hospital (US), Arkansas Children's Hospital (US), Nationwide Children's Hospital (US), St. Louis Children's Hospital (US), Primary Children's Hospital (US), Children's Mercy Hospital (US), Lucile Packard Children's Hospital (US), UF Health Shands Hospital (US), Children's Hospital of Philadelphia (US), Riley Hospital for Children (US), Dell Children's Medical Center of Central Texas (US), Kosair Children's Hospital (US), Children's Medical Center (US), Norton Hospital (US), Children's Hospital at Montefiore (US), Medical City Children's Hospital (US), University of Louisville Hospital (US), Stollery Children's Hospital (CA), Joe DiMaggio Children's Hospital (US), Children's Hospital of Richmond at VCU (US), Seattle University (US)
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Openalex Percentile: Top 11%
Muscle Physiology and Disorders
2.42
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